Equitable, timely and affordable access to therapies

We believe the entire drug access pathway must be streamlined and made more transparent and inclusive to address the needs of people living with ALS. This includes research and development, clinical trials, the process for new drug submission and approval, and public funding decisions.

The ALS community measures time not by months or years, but by loss – loss of function and loss of life. During a 180-day expedited timeline for Health Canada to review priority drugs, for example, 500 Canadians will die of ALS. Equitable, timely and affordable access to tomorrow’s therapies shouldn’t be a luxury – it should be a given.

How we advocate

There are more than 60 ALS drugs currently in the development pipeline and only two Health Canada-approved ALS therapies that have an impact on disease progression. Now is the time for government to streamline the drug access pathway and improve its transparency while creating meaningful involvement for those who know the realities of the disease best. In partnership with ALS Societies across Canada and coalition partners, we actively:

  • Work to help government understand the unintended implications associated with managing the rising cost of drugs, which could create an environment where innovative therapies do not come to Canada.
  • Advocate for timely, equitable and affordable access to clinically proven ALS therapies.
  • Give voice to how inconsistencies and gaps in the health care system are directly affecting Canadians living with ALS.

 

We urge the Government of Canada and the Government of Ontario to improve access to therapies, based on the following recommendations:

  • Create an environment that makes Canada a country of choice for industry to bring new therapies, from research and development, to clinical trials through to new drug submissions.
  • Coordinate, streamline and increase transparency of the regulatory processes, timelines, and transparency associated with bringing a drug to Canada in order to enable patients to better access drugs and inform decision-making processes.
  • The provinces, territories and federal government should collaborate to address the health care and funding inconsistencies across Canada that currently result in inequitable access to new therapies across the country.
  • The provincial government should implement a real plan that streamlines the process for newly-approved treatments moving through the Ontario Public Drug Program.

 

We have participated in the following consultations related to access to therapies:

In September 2018, we were invited to appear as a witness before the House of Commons Standing Committee on Health (HESA) related to access to therapies for rare diseases. Tammy Moore, ALS Canada CEO, spoke to the challenges faced by Canadians living with ALS related to accessing innovative and promising therapies before and after market approval.

In March 2019 HESA released its report Canadians Affected by Rare Diseases and Disorders: Improving Access to Treatment. The report acknowledged the experiences of people living with ALS and provided 19 recommendations to improve access to therapies.

Read the remarks here.

The development of a national pharmacare program has significant implications for all Canadians as it relates to access to therapies. There are also important considerations for the ALS community as we think about the treatment pipeline and the potential for new therapies to come to market in the coming years.

As a member of the Health Charities Coalition of Canada (HCCC), ALS Canada and members of the ALS community participated in the summer 2018 consultation process led by the federal government’s Advisory Council on the Implementation of National Pharmacare, chaired by Dr. Eric Hoskins. We also submitted a written response in the form of a letter as part of the consultation process. The letter was in support of the recommendations put forward by the Health Charities Coalition of Canada (HCCC), with additional considerations unique to the ALS community.

On June 12, 2019 the Advisory Council on the Implementation of National Pharmacare released its final report. Included in the report are recommendations on a strategy for expensive drugs for rare diseases and that patients should be involved in aspects of the decision-making process. These recommendations are a step in the right direction for equitable, timely, and affordable access to the proven ALS therapies of tomorrow and speak to how collectively our voices can have an impact.

Read ALS Canada’s Letter.

We were invited to participate in a webinar consultation led by the Expensive Drugs for Rare Diseases Working Group (EDRD WG) on a proposed supplemental process for the national and jurisdictional drug review process for complex/specialized drugs, including drugs for rare diseases. The discussion focused on a variety of topics including how to ensure the proposed supplemental process is transparent, streamlined and designed in consultation with patients and stakeholders.

Following the webinar, we provided a written submission response highlighting the perspectives of the ALS community as it relates to ensuring a streamlined and transparent drug access process.

Read the response.

In December 2017 the federal government proposed amendments to the Patented Medicines Regulations, the guidelines that govern how the Patented Medicine Prices Review Board (PMPRB) sets the price at which companies sell their drug to distributors across the country. The amendments are designed to create additional price determination factors for PMPRB, such as increasing the number of countries PMPRB compares drug prices to from seven to twelve countries.

The proposed changes to the regulations could affect the ability of Canadians living with ALS to access innovative and complex therapies in a timely and equitable manner, as well as create an even larger discrepancy between what therapies and clinical trials are available in Canada versus other countries. To draw attention to the potential implications for patients, as a member of the Health Charities Coalition of Canada (HCCC) ALS Canada submitted a letter highlighting key considerations for the proposed modernization of the Regulations.

Read ALS Canada’s Letter

Radicava (edaravone)

In October 2018, Health Canada approved edaravone for the treatment of ALS. Edaravone is the first drug to be approved by Health Canada for the treatment of ALS since the approval of riluzole in 2000. Approval from Health Canada means that edaravone can be marketed and sold in Canada.

What happens next

Other considerations, like the price of the drug and whether it will be covered through provincial drug plans, are separate steps in how drugs become approved and available to Canadians. The Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d’excellence en santé et en services sociaux (INESSS) in Quebec provide recommendations and advice to federal, provincial, and territorial drug plans regarding reimbursement decisions. Each publicly-funded drug program conducts an independent review of the therapy before ultimately deciding whether to cover the drug at no cost. The CADTH, Ontario Public Drug Program and B.C. PharmaCare processes include an opportunity for patient input.

How we advocate

Throughout the drug access process for edaravone, we have worked with ALS Societies across Canada to bring forward the experiences and perspectives of Canadians living with ALS.

In coordination with ALS Societies across Canada, we submitted a patient input submission to the Canadian Agency for Drugs and Technologies in Health (CADHT) as part of its clinical and economic review of Radicava (edaravone).

Read ALS Canada’s patient input submission.

In December 2018, we submitted a patient evidence submission to the Ontario Public Drug Program as part of its independent drug review process in order to make coverage decisions about edaravone. The content of the submission was based on the CADTH patient input submission, as well as a survey distributed to people living with ALS in Ontario who were accessing edaravone through the Mitsubishi Tanabe Pharma Corporation (MTPC) Edaravone Canadian Supply Programme.

Read the Ontario Public Drug Program patient evidence submission.